US20030215422A1
(en)
*
|
1996-09-11 |
2003-11-20 |
John A. Chiorini |
Aav4 vector and uses thereof
|
WO1999061601A2
(en)
*
|
1998-05-28 |
1999-12-02 |
The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services |
Aav5 vector and uses thereof
|
JP2004538005A
(ja)
|
2001-08-08 |
2004-12-24 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
シアル酸に結合するタンパク質を有するウイルスベクターの精製法
|
PL220644B1
(pl)
*
|
2001-11-13 |
2015-11-30 |
Univ Pennsylvania |
Wirus stowarzyszony z adenowirusem (AAV), kompozycja, wyizolowane białko kapsydowe, wyizolowane lub syntetyczne cząsteczki kwasu nukleinowego, sposób wytwarzania zrekombinowanego wirusa, komórka gospodarza
|
AU2013202568B2
(en)
*
|
2001-11-13 |
2015-09-17 |
The Trustees Of The University Of Pennsylvania |
Adeno-associated virus rh.8 (AAVrh.8) sequences and recombinant AAVs comprising same
|
ES2602352T3
(es)
*
|
2001-12-17 |
2017-02-20 |
The Trustees Of The University Of Pennsylvania |
Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
|
EP3339430A1
(de)
*
|
2001-12-17 |
2018-06-27 |
The Trustees of The University of Pennsylvania |
Adenoassoziierte virus(aav)-serotyp 9-sequenzen, vektoren damit und verwendungen davon
|
US7419817B2
(en)
*
|
2002-05-17 |
2008-09-02 |
The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. |
Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
|
AU2003304396A1
(en)
*
|
2002-06-28 |
2005-02-25 |
University Of Florida Research Foundation, Inc. |
Raav compositions and methods for delivery of human factor vii polypeptides and treatment of hemophilia a
|
WO2004108922A2
(en)
*
|
2003-04-25 |
2004-12-16 |
The Trustees Of The University Of Pennsylvania |
Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
|
US8927269B2
(en)
|
2003-05-19 |
2015-01-06 |
The United States Of America, As Represented By The Secretary, Department Of Health And Human Services |
Avian adenoassociated virus and uses thereof
|
WO2004110483A1
(en)
*
|
2003-05-31 |
2004-12-23 |
Yiyou Chen |
Method and composition of a novel vaccine design for the prevention and treatment of sars
|
US7186699B2
(en)
*
|
2003-06-03 |
2007-03-06 |
Cell Genesys, Inc. |
Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
|
US7906111B2
(en)
*
|
2003-09-30 |
2011-03-15 |
The Trustees Of The University Of Pennsylvania |
Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
|
WO2005049850A2
(en)
|
2003-11-14 |
2005-06-02 |
University Of Washington |
Compositions and methods for systemic nucleic acid sequence delivery
|
US8137960B2
(en)
|
2003-12-04 |
2012-03-20 |
The United States Of America As Represented By The Department Of Health And Human Services |
Bovine adeno-associated viral (BAAV) vector and uses thereof
|
US20060078542A1
(en)
*
|
2004-02-10 |
2006-04-13 |
Mah Cathryn S |
Gel-based delivery of recombinant adeno-associated virus vectors
|
CA2563500C
(en)
|
2004-04-28 |
2016-06-28 |
The Trustees Of The University Of Pennsylvania |
Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
|
ATE497785T1
(de)
|
2004-04-28 |
2011-02-15 |
Univ Pennsylvania |
Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben
|
JP2008500364A
(ja)
*
|
2004-05-25 |
2008-01-10 |
キメラコア, インコーポレイテッド |
自己集合性ナノ粒子薬物送達システム
|
US20080188431A1
(en)
*
|
2004-09-08 |
2008-08-07 |
Chiorini John A |
Transcytosis of Adeno-Associated Viruses
|
WO2006119432A2
(en)
*
|
2005-04-29 |
2006-11-09 |
The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services |
Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
|
CN105770909A
(zh)
*
|
2005-05-02 |
2016-07-20 |
建新公司 |
脊髓失调症的基因治疗
|
PT3520823T
(pt)
|
2005-05-02 |
2021-09-16 |
Genzyme Corp |
Terapia genética para distúrbios neurometabólicos
|
EP2007795B1
(de)
|
2006-03-30 |
2016-11-16 |
The Board Of Trustees Of The Leland Stanford Junior University |
Aav-capsid-proteine
|
CA2648581A1
(en)
*
|
2006-04-07 |
2008-09-12 |
Chimeros, Inc. |
Compositions and methods for treating b- cell malignancies
|
CN101495624A
(zh)
*
|
2006-04-28 |
2009-07-29 |
宾夕法尼亚州立大学托管会 |
衣壳免疫原性降低的经修饰aav载体及其用途
|
JP5268890B2
(ja)
|
2006-04-28 |
2013-08-21 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
Aavの規模適応性の製造方法
|
WO2008124165A2
(en)
*
|
2007-04-09 |
2008-10-16 |
Chimeros, Inc. |
Self-assembling nanoparticle drug delivery system
|
US9217155B2
(en)
|
2008-05-28 |
2015-12-22 |
University Of Massachusetts |
Isolation of novel AAV'S and uses thereof
|
US8263601B2
(en)
|
2009-02-27 |
2012-09-11 |
Concert Pharmaceuticals, Inc. |
Deuterium substituted xanthine derivatives
|
WO2010120874A2
(en)
|
2009-04-14 |
2010-10-21 |
Chimeros, Inc. |
Chimeric therapeutics, compositions, and methods for using same
|
WO2010138263A2
(en)
|
2009-05-28 |
2010-12-02 |
University Of Massachusetts |
Novel aav 's and uses thereof
|
WO2011032100A1
(en)
|
2009-09-11 |
2011-03-17 |
Government Of The U.S.A., As Represented By The Secretary, Department Of Health And Human Services |
Inhibitors of kshv vil6 and human il6
|
US20120244127A1
(en)
|
2009-10-01 |
2012-09-27 |
The Trustees Of The University Of Pennsylvania |
AAV Vectors Expressing SEC10 for Treating Kidney Damage
|
SG10201502270TA
(en)
|
2010-03-29 |
2015-05-28 |
Univ Pennsylvania |
Pharmacologically induced transgene ablation system
|
US9315825B2
(en)
|
2010-03-29 |
2016-04-19 |
The Trustees Of The University Of Pennsylvania |
Pharmacologically induced transgene ablation system
|
WO2011133874A1
(en)
|
2010-04-23 |
2011-10-27 |
University Of Massachusetts |
Multicistronic expression constructs
|
EP2826860B1
(de)
|
2010-04-23 |
2018-08-22 |
University of Massachusetts |
Auf das ZNS abzielende AAV Vektoren und Verfahren zur Verwendung davon
|
EP2561075B1
(de)
|
2010-04-23 |
2018-06-27 |
University of Massachusetts |
Aav-basierte behandlung von cholesterinerkrankungen
|
US9309534B2
(en)
|
2010-07-12 |
2016-04-12 |
Universidad Autonoma De Barcelona |
Gene therapy composition for use in diabetes treatment
|
WO2012075040A2
(en)
*
|
2010-11-30 |
2012-06-07 |
Shire Human Genetic Therapies, Inc. |
mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
|
US10392632B2
(en)
|
2011-02-14 |
2019-08-27 |
The Children's Hospital Of Philadelphia |
AAV8 vector with enhanced functional activity and methods of use thereof
|
EP3699286A1
(de)
|
2011-04-20 |
2020-08-26 |
The Trustees of the University of Pennsylvania |
Dosierpläne und zusammensetzungen zur aav-vermittelten passiven immunisierung von luftbürtigen krankheitserregern
|
US9289477B2
(en)
|
2011-04-29 |
2016-03-22 |
Selecta Biosciences, Inc. |
Tolerogenic synthetic nanocarriers to reduce cytotoxic T lymphocyte responses
|
AU2012305714A1
(en)
|
2011-09-09 |
2014-03-27 |
Biomed Realty, L.P. |
Methods and compositions for controlling assembly of viral proteins
|
CN110777124A
(zh)
|
2011-10-27 |
2020-02-11 |
威尔斯达眼科制剂公司 |
编码视杆细胞来源的视锥细胞活力因子的载体
|
WO2013078316A1
(en)
|
2011-11-23 |
2013-05-30 |
Nationwide Children's Hospital, Inc. |
Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
|
DK2814513T3
(en)
*
|
2012-02-14 |
2018-02-26 |
Univ California |
Systemic administration and regulated expression of paracrine for cardiovascular disease and other conditions
|
JP5926580B2
(ja)
|
2012-03-01 |
2016-05-25 |
ユー・ディー・シー アイルランド リミテッド |
有機電界発光素子、有機電界発光素子用材料、並びに、該素子を用いた発光装置、表示装置、照明装置及び該素子に用いられる化合物
|
EP2872183B1
(de)
|
2012-07-11 |
2018-09-26 |
The Trustees Of The University Of Pennsylvania |
Aav-vermittelte gentherapie zur x-chromosomalen netzhautdegeneration
|
EP2692868A1
(de)
|
2012-08-02 |
2014-02-05 |
Universitat Autònoma De Barcelona |
Adenoassoziierte virale (AAV) Vektoren für die Transduktion von Fettgewebe
|
PL2900686T3
(pl)
*
|
2012-09-28 |
2021-01-25 |
The University Of North Carolina At Chapel Hill |
Wektory aav ukierunkowane na oligodendrocyty
|
US9567376B2
(en)
|
2013-02-08 |
2017-02-14 |
The Trustees Of The University Of Pennsylvania |
Enhanced AAV-mediated gene transfer for retinal therapies
|
US10266845B2
(en)
|
2013-02-08 |
2019-04-23 |
The Trustees Of The University Of Pennsylvania |
Enhanced AAV-mediated gene transfer for retinal therapies
|
HRP20231183T1
(hr)
|
2013-02-15 |
2024-01-05 |
Bioverativ Therapeutics Inc. |
Optimizirani gen faktora viii
|
US8957044B2
(en)
|
2013-03-01 |
2015-02-17 |
Wake Forest University Health Sciences |
Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
|
SG11201507507PA
(en)
*
|
2013-03-15 |
2015-10-29 |
Univ Pennsylvania |
Compositions and methods for treating mpsi
|
CN109652385A
(zh)
|
2013-04-20 |
2019-04-19 |
全国儿童医院研究所 |
外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
|
US9719106B2
(en)
|
2013-04-29 |
2017-08-01 |
The Trustees Of The University Of Pennsylvania |
Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
|
PE20160188A1
(es)
*
|
2013-07-22 |
2016-04-27 |
Philadelphia Children Hospital |
Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos
|
EP3690044B1
(de)
|
2014-02-11 |
2024-01-10 |
The Regents of the University of Colorado, a body corporate |
Crispr-aktiviertes multiplexed-genom-engineering
|
GB201403684D0
(en)
|
2014-03-03 |
2014-04-16 |
King S College London |
Vector
|
BR122023023004A2
(pt)
|
2014-03-09 |
2023-12-26 |
The Trustees Of The University Of Pennsylvania |
Vetores virais recombinantes, vírus adeno-associado recombinante, composição farmacêutica, bem como uso dos mesmos
|
EP2933335A1
(de)
|
2014-04-18 |
2015-10-21 |
Genethon |
Verfahren zur Behandlung peripherer Neuropathien und Motoneuronerkrankungen
|
WO2015164723A1
(en)
|
2014-04-25 |
2015-10-29 |
The Trustees Of The University Of Pennsylvania |
Methods and compositions for treating metastatic breast cancer and other cancers in the brain
|
ES2876409T3
(es)
|
2014-04-25 |
2021-11-12 |
Univ Pennsylvania |
Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
|
PT3142750T
(pt)
|
2014-05-13 |
2020-09-22 |
Univ Pennsylvania |
Composições que compreendem aav expressando construções de anticorpos duplos e suas utilizações
|
WO2015191508A1
(en)
|
2014-06-09 |
2015-12-17 |
Voyager Therapeutics, Inc. |
Chimeric capsids
|
EP2960336A1
(de)
|
2014-06-27 |
2015-12-30 |
Genethon |
Effiziente systemische Behandlung von Muskeldystrophie
|
EP3160478A4
(de)
|
2014-06-30 |
2018-05-16 |
Bioverativ Therapeutics Inc. |
Optimiertes faktor-ix-gen
|
EP2975116B1
(de)
*
|
2014-07-16 |
2019-08-21 |
Max-Delbrück-Centrum für Molekulare Medizin |
Tgif2-induzierte Neuprogrammierung von Leberzellen zu Pankreas-Vorläuferzellen und medizinische Verwendungen davon
|
US11053494B2
(en)
|
2014-08-09 |
2021-07-06 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene
|
WO2016037163A1
(en)
|
2014-09-07 |
2016-03-10 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses
|
WO2016041588A1
(en)
*
|
2014-09-16 |
2016-03-24 |
Universitat Autònoma De Barcelona |
Adeno-associated viral vectors for the gene therapy of metabolic diseases
|
US10370432B2
(en)
|
2014-10-03 |
2019-08-06 |
University Of Massachusetts |
Heterologous targeting peptide grafted AAVS
|
US10711270B2
(en)
|
2014-10-03 |
2020-07-14 |
University Of Massachusetts |
High efficiency library-identified AAV vectors
|
CN107073051B
(zh)
|
2014-10-21 |
2021-08-24 |
马萨诸塞大学 |
重组aav变体及其用途
|
EP3215191A4
(de)
|
2014-11-05 |
2018-08-01 |
Voyager Therapeutics, Inc. |
Aadc-polynukleotide zur behandlung von morbus parkinson
|
CN107109407A
(zh)
|
2014-11-14 |
2017-08-29 |
沃雅戈治疗公司 |
治疗肌萎缩性侧索硬化(als)的组合物和方法
|
SG11201703419UA
(en)
|
2014-11-14 |
2017-05-30 |
Voyager Therapeutics Inc |
Modulatory polynucleotides
|
US11697825B2
(en)
|
2014-12-12 |
2023-07-11 |
Voyager Therapeutics, Inc. |
Compositions and methods for the production of scAAV
|
JP6754361B2
(ja)
|
2014-12-16 |
2020-09-09 |
ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ |
若年型バッテン病のための遺伝子療法
|
EP3978614A3
(de)
|
2015-01-07 |
2022-07-27 |
Universitat Autònoma de Barcelona |
Einzelvektor-genkonstrukt mit insulin und glucokinasegenen
|
CA2970730A1
(en)
|
2015-01-16 |
2016-07-21 |
Voyager Therapeutics, Inc. |
Central nervous system targeting polynucleotides
|
WO2016130600A2
(en)
|
2015-02-09 |
2016-08-18 |
Duke University |
Compositions and methods for epigenome editing
|
EP3256170B1
(de)
|
2015-02-13 |
2020-09-23 |
University of Massachusetts |
Zusammensetzungen und verfahren zur transitorischen freisetzung von polypeptiden
|
CN108093639B
(zh)
|
2015-04-16 |
2022-07-19 |
埃默里大学 |
用于肝脏中蛋白质表达的重组启动子和载体及其用途
|
EP3285780A4
(de)
|
2015-04-24 |
2018-12-19 |
University of Massachusetts |
Modifizierte aav-konstruktionen und verwendungen davon
|
WO2016176191A1
(en)
|
2015-04-27 |
2016-11-03 |
The Trustees Of The University Of Pennsylvania |
Dual aav vector system for crispr/cas9 mediated correction of human disease
|
GB201508026D0
(en)
|
2015-05-11 |
2015-06-24 |
Ucl Business Plc |
Capsid
|
US11535665B2
(en)
|
2015-05-13 |
2022-12-27 |
The Trustees Of The University Of Pennsylvania |
AAV-mediated expression of anti-influenza antibodies and methods of use thereof
|
PE20180675A1
(es)
|
2015-06-23 |
2018-04-19 |
Childrens Hospital Philadelphia |
Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos
|
US10676735B2
(en)
|
2015-07-22 |
2020-06-09 |
Duke University |
High-throughput screening of regulatory element function with epigenome editing technologies
|
ES2929110T3
(es)
|
2015-08-25 |
2022-11-24 |
Univ Duke |
Composiciones y métodos para mejorar la especificidad en ingeniería genética usando endonucleasas guiadas por ARN
|
KR20180057636A
(ko)
|
2015-08-31 |
2018-05-30 |
더 트러스티스 오브 더 유니버시티 오브 펜실바니아 |
반려 동물 치료용 aav-epo
|
CA2999299A1
(en)
|
2015-09-24 |
2017-03-30 |
The Trustees Of The University Of Pennsylvania |
Composition and method for treating complement-mediated disease
|
IL292830B2
(en)
|
2015-09-28 |
2023-06-01 |
Univ Florida |
Methods and compositions for stealth virus antibody vectors
|
US11273227B2
(en)
|
2015-10-09 |
2022-03-15 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods useful in treating Stargardt's disease and other ocular disorders
|
EP4089175A1
(de)
|
2015-10-13 |
2022-11-16 |
Duke University |
Genom-engineering mit typ-i-crispr-systemen in eukaryotischen zellen
|
WO2017066764A2
(en)
*
|
2015-10-16 |
2017-04-20 |
William Marsh Rice University |
Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
|
WO2017070525A1
(en)
|
2015-10-22 |
2017-04-27 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance in neurodegenerative disease
|
EP3364996B1
(de)
|
2015-10-22 |
2021-08-25 |
University of Massachusetts |
Gegen die prostata gerichtete adeno-assoziierte virusserotypvektoren
|
US11135313B2
(en)
|
2015-10-28 |
2021-10-05 |
The Trustees Of The University Of Pennsylvania |
Intrathecal administration of adeno-associated-viral vectors for gene therapy
|
US20180230489A1
(en)
|
2015-10-28 |
2018-08-16 |
Voyager Therapeutics, Inc. |
Regulatable expression using adeno-associated virus (aav)
|
AU2016354550B2
(en)
|
2015-11-13 |
2020-04-16 |
Takeda Pharmaceutical Company Limited |
Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
|
EA038288B1
(ru)
|
2015-11-13 |
2021-08-05 |
Такеда Фармасьютикал Компани Лимитед |
Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
|
AU2016362477A1
(en)
*
|
2015-12-02 |
2018-06-14 |
Voyager Therapeutics, Inc. |
Assays for the detection of AAV neutralizing antibodies
|
US10406244B2
(en)
|
2015-12-02 |
2019-09-10 |
The Board Of Trustees Of The Leland Stanford Junior University |
AAV vectors with expanded packaging capacity
|
EP3384034B1
(de)
|
2015-12-02 |
2020-07-08 |
The Board of Trustees of the Leland Stanford Junior University |
Neue rekombinante kapside des adeno-associated virus mit erhöhtem tropismus für menschliche skelett- muskulatur
|
FR3044926B1
(fr)
*
|
2015-12-09 |
2020-01-31 |
Genethon |
Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
|
EP3400304B1
(de)
*
|
2015-12-11 |
2022-04-06 |
The Trustees Of The University Of Pennsylvania |
Gentherapeutische behandlung der familiären hypercholesterinämie
|
US11015173B2
(en)
|
2015-12-11 |
2021-05-25 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for AAV1
|
EP3387118B1
(de)
|
2015-12-11 |
2022-04-06 |
The Trustees Of The University Of Pennsylvania |
Skalierbares reinigungsverfahren für aavrh10
|
WO2017100676A1
(en)
|
2015-12-11 |
2017-06-15 |
The Trustees Of The University Of Pennsylvania |
Scalable purification method for aav8
|
EP3387138B1
(de)
|
2015-12-11 |
2022-01-26 |
The Trustees Of The University Of Pennsylvania |
Skalierbares reinigungsverfahren für aav9
|
CN109069668B
(zh)
|
2015-12-14 |
2023-04-18 |
宾夕法尼亚州大学信托人 |
用于眼病的基因疗法
|
CA3007330A1
(en)
|
2015-12-14 |
2017-06-22 |
The Trustees Of The University Of Pennsylvania |
Composition for treatment of crigler-najjar syndrome
|
SI3411478T1
(sl)
|
2016-02-01 |
2022-10-28 |
Bioverativ Therapeutics Inc. |
Optimirani geni dejavnika VIII
|
WO2017136536A1
(en)
|
2016-02-02 |
2017-08-10 |
University Of Massachusetts |
Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
|
MA43968A
(fr)
|
2016-02-03 |
2018-12-12 |
Univ Pennsylvania |
Thérapie génique pour traiter la mucopolysaccharidose de type i
|
US10179176B2
(en)
|
2016-02-16 |
2019-01-15 |
The Board Of Trustees Of The Leland Stanford Junior University |
Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
|
US11345913B2
(en)
|
2016-04-02 |
2022-05-31 |
Research Institute At Nationwide Children's Hospital |
Modified U6 promoter system for tissue specific expression
|
US11413356B2
(en)
|
2016-04-15 |
2022-08-16 |
University Of Massachusetts |
Methods and compositions for treating metabolic imbalance
|
SG11201808812RA
(en)
*
|
2016-04-15 |
2018-11-29 |
Univ Pennsylvania |
Novel aav8 mutant capsids and compositions containing same
|
PE20181922A1
(es)
|
2016-04-15 |
2018-12-11 |
Univ Pennsylvania |
Terapia genica para tratar la hemofilia a
|
MA44873A
(fr)
|
2016-04-15 |
2019-03-13 |
Univ Pennsylvania |
Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative
|
MA45477A
(fr)
|
2016-04-15 |
2019-02-20 |
Res Inst Nationwide Childrens Hospital |
Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
|
IL262211B2
(en)
|
2016-04-15 |
2024-01-01 |
Univ Pennsylvania |
Gene therapy for the treatment of type II mucositis
|
US11401527B2
(en)
|
2016-04-17 |
2022-08-02 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods useful for prophylaxis of organophosphates
|
WO2017189959A1
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
WO2017189964A2
(en)
|
2016-04-29 |
2017-11-02 |
Voyager Therapeutics, Inc. |
Compositions for the treatment of disease
|
ES2941502T3
(es)
|
2016-05-13 |
2023-05-23 |
4D Molecular Therapeutics Inc |
Variantes de cápsides de virus adenoasociado y procedimientos de utilización de las mismas
|
CN110214187B
(zh)
|
2016-05-18 |
2024-01-30 |
沃雅戈治疗公司 |
调节性多核苷酸
|
CA3024449A1
(en)
|
2016-05-18 |
2017-11-23 |
Voyager Therapeutics, Inc. |
Compositions and methods of treating huntington's disease
|
WO2017218852A1
(en)
|
2016-06-15 |
2017-12-21 |
University Of Massachusetts |
Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
|
US11471462B2
(en)
|
2016-06-27 |
2022-10-18 |
The Broad Institute, Inc. |
Compositions and methods for detecting and treating diabetes
|
RU2764920C2
(ru)
|
2016-07-08 |
2022-01-24 |
Зе Трастис Оф Зе Юниверсити Оф Пенсильвания |
Способы и композиции для лечения нарушений и заболеваний, связанных с rdh12
|
US11883470B2
(en)
|
2016-07-25 |
2024-01-30 |
The Trustees Of The University Of Pennsylvania |
Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
|
US11298041B2
(en)
|
2016-08-30 |
2022-04-12 |
The Regents Of The University Of California |
Methods for biomedical targeting and delivery and devices and systems for practicing the same
|
WO2018071831A1
(en)
|
2016-10-13 |
2018-04-19 |
University Of Massachusetts |
Aav capsid designs
|
EP3548065B1
(de)
|
2016-12-01 |
2022-11-09 |
INSERM - Institut National de la Santé et de la Recherche Médicale |
Pharmazeutische zusammensetzungen zur behandlung von degenerativen netzhauterkrankungen
|
EP3562494A4
(de)
*
|
2016-12-30 |
2020-08-19 |
The Trustees Of The University Of Pennsylvania |
Gentherapie zur behandlung von phenylketonurie
|
KR20230160411A
(ko)
*
|
2016-12-30 |
2023-11-23 |
더 트러스티스 오브 더 유니버시티 오브 펜실베니아 |
윌슨병을 치료하기 위한 유전자 치료
|
AU2018205496A1
(en)
|
2017-01-07 |
2019-07-25 |
Selecta Biosciences, Inc. |
Patterned dosing of immunosuppressants coupled to synthetic nanocarriers
|
US11535866B2
(en)
|
2017-02-01 |
2022-12-27 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating citrullenemia
|
JP2020510648A
(ja)
|
2017-02-20 |
2020-04-09 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
家族性高コレステロール血症を処置するための遺伝子治療
|
KR20190135000A
(ko)
|
2017-02-28 |
2019-12-05 |
더 트러스티스 오브 더 유니버시티 오브 펜실바니아 |
Aav 벡터를 기반으로 하는 인플루엔자 백신
|
SI3589730T1
(sl)
|
2017-02-28 |
2024-04-30 |
The Trustees Of The University Of Pennsylvania |
Adeno-povezan virus (AAV) clade F vektor in njihova uporaba
|
JOP20190200A1
(ar)
|
2017-02-28 |
2019-08-27 |
Univ Pennsylvania |
تركيبات نافعة في معالجة ضمور العضل النخاعي
|
KR20230093072A
(ko)
|
2017-03-01 |
2023-06-26 |
더 트러스티스 오브 더 유니버시티 오브 펜실베니아 |
안구 장애에 대한 유전자 치료
|
EP4245852A3
(de)
|
2017-03-17 |
2023-11-22 |
Research Institute at Nationwide Children's Hospital |
Adeno-assoziierte virusvektorverabreichung von muskelspezifischem mikrodystrophin zur behandlung von muskeldystrophie
|
WO2019012336A2
(en)
|
2017-03-17 |
2019-01-17 |
Newcastle University |
ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
|
JP7291398B2
(ja)
|
2017-03-30 |
2023-06-15 |
ザ ユニバーシティー オブ クイーンズランド |
キメラ分子およびその使用
|
EP3610016A1
(de)
|
2017-04-10 |
2020-02-19 |
Genethon |
Antisense-targeting-dynamin 2 und verwendung zur behandlung von zentronukleären myopathien und neuropathien
|
AU2018253303A1
(en)
|
2017-04-14 |
2019-10-31 |
Regenxbio Inc. |
Treatment of mucopolysaccharidosis II with recombinant human iduronate-2 sulfatase (IDS) produced by human neural or glial cells
|
IL301059A
(en)
|
2017-04-21 |
2023-05-01 |
Prec Biosciences Inc |
Transgenic magnonucleases specific for recognition sequences in the PCSK9 gene
|
WO2018200542A1
(en)
|
2017-04-24 |
2018-11-01 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for ocular disorders
|
SG11201909868YA
(en)
|
2017-05-05 |
2019-11-28 |
Voyager Therapeutics Inc |
Compositions and methods of treating huntington's disease
|
EP3618839A4
(de)
|
2017-05-05 |
2021-06-09 |
Voyager Therapeutics, Inc. |
Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als)
|
US11633504B2
(en)
|
2017-05-09 |
2023-04-25 |
Emory University |
Nucleic acids encoding clotting factor variants and their use
|
CA3061655A1
(en)
|
2017-05-11 |
2018-11-15 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for neuronal ceroid lipofuscinoses
|
WO2018213618A1
(en)
|
2017-05-17 |
2018-11-22 |
The General Hospital Corporation |
Gene therapy for tuberous sclerosis
|
KR102551733B1
(ko)
|
2017-05-22 |
2023-07-06 |
다케다 야쿠힌 고교 가부시키가이샤 |
B형 혈우병 유전자 요법을 위한 증가된 발현을 가지는 재조합 fix 변이체를 암호화하는 바이러스 벡터
|
US11793887B2
(en)
|
2017-05-31 |
2023-10-24 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for treating peroxisomal disorders
|
WO2018232149A1
(en)
|
2017-06-14 |
2018-12-20 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for ocular disorders
|
JOP20190269A1
(ar)
|
2017-06-15 |
2019-11-20 |
Voyager Therapeutics Inc |
بولي نوكليوتيدات aadc لعلاج مرض باركنسون
|
SG11201912631PA
(en)
|
2017-07-06 |
2020-01-30 |
Univ Pennsylvania |
Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
|
WO2019008157A1
(en)
|
2017-07-07 |
2019-01-10 |
Genethon |
NOVEL POLYNUCLEOTIDES ENCODING HUMAN FKRP PROTEIN
|
CN111132626B
(zh)
|
2017-07-17 |
2024-01-30 |
沃雅戈治疗公司 |
轨迹阵列引导系统
|
WO2019028306A2
(en)
|
2017-08-03 |
2019-02-07 |
Voyager Therapeutics, Inc. |
COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES
|
FI3684423T3
(fi)
|
2017-09-20 |
2023-06-15 |
4D Molecular Therapeutics Inc |
Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
|
BR112020005249A2
(pt)
|
2017-09-22 |
2020-09-24 |
The Trustees Of The University Of Pennsylvania |
terapia de gene para tratar mucopolissacaridose tipo ii
|
AU2018347583A1
(en)
|
2017-10-13 |
2020-05-21 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating anti-viral transfer vector IgM responses
|
WO2019079242A1
(en)
|
2017-10-16 |
2019-04-25 |
Voyager Therapeutics, Inc. |
TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
|
AU2018352236A1
(en)
|
2017-10-16 |
2020-04-23 |
The Curators Of The University Of Missouri |
Treatment of amyotrophic lateral sclerosis (ALS)
|
JP2021501196A
(ja)
|
2017-10-18 |
2021-01-14 |
リジェネックスバイオ インコーポレイテッド |
完全ヒト翻訳後修飾抗体による治療剤
|
JP7213238B2
(ja)
|
2017-10-18 |
2023-01-26 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達
|
AU2018350990A1
(en)
|
2017-10-18 |
2020-05-21 |
Regenxbio Inc. |
Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
|
CA3079416A1
(en)
|
2017-10-20 |
2019-04-25 |
Research Institute At Nationwide Children's Hospital |
Methods and materials for nt-3 gene therapy
|
PE20210915A1
(es)
|
2017-11-27 |
2021-05-19 |
4D Molecular Therapeutics Inc |
Capsides variantes de virus adenoasociados y su uso para inhibir la angiogenesis
|
AU2018375163A1
(en)
|
2017-11-30 |
2020-06-11 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for mucopolysaccharidosis IIIB
|
CA3083416A1
(en)
|
2017-11-30 |
2019-06-06 |
The Trustees Of The University Of Pennsylvania |
Gene therapy for mucopolysaccharidosis iii a
|
EA202091509A1
(ru)
|
2017-12-19 |
2020-09-22 |
Акуос, Инк. |
Aav-опосредованная доставка терапевтических антител во внутреннее ухо
|
JP2021511020A
(ja)
|
2018-01-17 |
2021-05-06 |
アドリーナス セラピューティクス, インコーポレイテッド |
21−ヒドロキシラーゼ欠損症のためのアデノ随伴ウイルス遺伝子療法
|
CA3090226A1
(en)
*
|
2018-02-01 |
2019-08-08 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
|
US10610606B2
(en)
|
2018-02-01 |
2020-04-07 |
Homology Medicines, Inc. |
Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
|
EP3755795A4
(de)
|
2018-02-19 |
2022-07-20 |
Homology Medicines, Inc. |
Adeno-assoziierte viruszusammensetzungen zur wiederherstellung der f8-genfunktion und verfahren zur verwendung davon
|
CN112384204B
(zh)
|
2018-02-26 |
2023-03-14 |
安托瑞斯公司 |
致耐受性脂质体及其使用方法
|
WO2019183605A1
(en)
*
|
2018-03-23 |
2019-09-26 |
University Of Massachusetts |
Gene therapeutics for treating bone disorders
|
US11608510B2
(en)
|
2018-03-30 |
2023-03-21 |
The Board Of Trustees Of The Leland Stanford Junior University |
Recombinant adeno-associated virus capsids with enhanced human pancreatic tropism
|
WO2019195449A1
(en)
|
2018-04-03 |
2019-10-10 |
Stridebio, Inc. |
Antibody-evading virus vectors
|
CA3098565A1
(en)
|
2018-04-29 |
2019-11-07 |
Claire G. ZHANG |
Scalable clarification process for recombinant aav production
|
CA3098566A1
(en)
|
2018-04-29 |
2019-11-07 |
Zhuchun WU |
Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
|
CA3102840A1
(en)
|
2018-06-05 |
2019-12-12 |
Lifeedit, Inc. |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
WO2019241486A1
(en)
|
2018-06-13 |
2019-12-19 |
Voyager Therapeutics, Inc. |
Engineered 5' untranslated regions (5' utr) for aav production
|
CN112469822A
(zh)
|
2018-06-14 |
2021-03-09 |
再生生物股份有限公司 |
用于重组aav生产的阴离子交换色谱
|
BR112020025995A2
(pt)
|
2018-06-18 |
2021-03-23 |
Research Institute At Nationwide Children's Hospital |
administração de microdistrofina músculo-específica por vetor de vírus adeno-associado para tratar a distrofia muscular
|
GB201811368D0
(en)
|
2018-07-11 |
2018-08-29 |
Ucb Biopharma Sprl |
Antibody
|
MX2021000637A
(es)
|
2018-07-16 |
2021-06-23 |
Selecta Biosciences Inc |
Métodos y composiciones de vectores y constructos ornitina transcarbamilasa (otc).
|
EP3823676A1
(de)
|
2018-07-16 |
2021-05-26 |
Selecta Biosciences, Inc. |
Verfahren und zusammensetzungen von mma-konstrukten und vektoren
|
AU2019310459A1
(en)
|
2018-07-24 |
2021-02-18 |
Voyager Therapeutics, Inc. |
Systems and methods for producing gene therapy formulations
|
CA3108113A1
(en)
|
2018-08-10 |
2020-02-13 |
Michael GILLMEISTER |
Scalable method for recombinant aav production
|
US20210324417A1
(en)
|
2018-08-29 |
2021-10-21 |
Research Institute At Nationwide Children's Hospital |
Products and methods for inhibition of expression of mutant gars protein
|
KR20210102870A
(ko)
|
2018-08-30 |
2021-08-20 |
테나야 테라퓨틱스, 인코포레이티드 |
미오카르딘 및 ascl1을 사용한 심장 세포 재프로그래밍
|
WO2020072849A1
(en)
|
2018-10-04 |
2020-04-09 |
Voyager Therapeutics, Inc. |
Methods for measuring the titer and potency of viral vector particles
|
WO2020072844A1
(en)
|
2018-10-05 |
2020-04-09 |
Voyager Therapeutics, Inc. |
Engineered nucleic acid constructs encoding aav production proteins
|
UY38407A
(es)
|
2018-10-15 |
2020-05-29 |
Novartis Ag |
Anticuerpos estabilizadores de trem2
|
CN113166781A
(zh)
|
2018-10-15 |
2021-07-23 |
沃雅戈治疗公司 |
在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
|
EP3867412A1
(de)
|
2018-10-15 |
2021-08-25 |
REGENXBIO Inc. |
Verfahren zur messung der infektiosität von replikationsdefektiven viralen vektoren und viren
|
CN113302291A
(zh)
|
2018-10-22 |
2021-08-24 |
罗切斯特大学 |
使用逆转录病毒整合酶-Cas9融合蛋白通过定向非同源DNA插入进行的基因组编辑
|
US20220145296A1
(en)
|
2018-12-27 |
2022-05-12 |
LifeEDIT Therapeutics, Inc. |
Polypeptides useful for gene editing and methods of use
|
US20220089670A1
(en)
|
2018-12-28 |
2022-03-24 |
University Of Rochester |
Gene Therapy for BEST1 Dominant Mutations
|
US20220106592A1
(en)
|
2018-12-31 |
2022-04-07 |
Research Institute At Nationwide Children's Hospital |
DUX4 RNA Silencing Using RNA Targeting CRISPR-CAS13b
|
MX2021008135A
(es)
|
2019-01-04 |
2021-08-11 |
Ultragenyx Pharmaceutical Inc |
Construcciones de terapia genica para el tratamiento de la enfermedad de wilson.
|
CN113544267A
(zh)
|
2019-01-14 |
2021-10-22 |
罗切斯特大学 |
使用CRISPR-Cas进行靶向核RNA裂解和聚腺苷酸化
|
TW202039546A
(zh)
|
2019-01-16 |
2020-11-01 |
美商巴克斯歐塔公司 |
用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
|
CN109628490A
(zh)
*
|
2019-01-26 |
2019-04-16 |
青岛农业大学 |
一种预防犬瘟热的shRNA重组腺相关病毒
|
WO2020160508A1
(en)
|
2019-01-31 |
2020-08-06 |
Oregon Health & Science University |
Methods for using transcription-dependent directed evolution of aav capsids
|
SG11202108044YA
(en)
|
2019-02-25 |
2021-09-29 |
Novartis Ag |
Compositions and methods to treat bietti crystalline dystrophy
|
CN113474461A
(zh)
|
2019-02-25 |
2021-10-01 |
诺华股份有限公司 |
治疗bietti晶体营养不良的组合物和方法
|
AR118465A1
(es)
|
2019-03-21 |
2021-10-06 |
Stridebio Inc |
Vectores de virus adenoasociados recombinantes
|
JP2022526526A
(ja)
|
2019-03-25 |
2022-05-25 |
ジェネトン |
オーバーラップaavベクターを使用する大きいサイズのクアシジストロフィンの産生
|
US20220143221A1
(en)
|
2019-04-03 |
2022-05-12 |
Regenxbio, Inc. |
Gene Therapy For Eye Pathologies
|
TW202102526A
(zh)
|
2019-04-04 |
2021-01-16 |
美商銳進科斯生物股份有限公司 |
重組腺相關病毒及其用途
|
HUE064411T2
(hu)
|
2019-04-11 |
2024-03-28 |
Regenxbio Inc |
Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
|
EP4272817A3
(de)
|
2019-04-19 |
2024-01-24 |
RegenxBio Inc. |
Adeno-assoziierte virusvektorformulierungen und verfahren
|
AU2020262416A1
(en)
|
2019-04-24 |
2021-12-16 |
Regenxbio Inc. |
Fully-human post-translationally modified antibody therapeutics
|
AU2020265215A1
(en)
|
2019-04-28 |
2021-11-18 |
Selecta Biosciences, Inc. |
Methods for treatment of subjects with preexisting immunity to viral transfer vectors
|
WO2020243261A1
(en)
|
2019-05-28 |
2020-12-03 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuated anti-viral transfer vector immune response
|
KR20220069917A
(ko)
|
2019-07-02 |
2022-05-27 |
엠6피 테라퓨틱스 (스위처랜드) 엘엘씨 |
리소좀 축적 장애의 치료를 위한 벡터 조성물 및 이의 사용 방법
|
WO2021005223A1
(en)
|
2019-07-10 |
2021-01-14 |
INSERM (Institut National de la Santé et de la Recherche Médicale) |
Methods for the treatment of epilepsy
|
US20230137971A1
(en)
|
2019-07-11 |
2023-05-04 |
Tenaya Therapeutics Inc. |
Cardiac cell reprogramming with micrornas and other factors
|
US10801042B1
(en)
|
2019-07-15 |
2020-10-13 |
Vigene Biosciences, Inc. |
Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
|
US10653731B1
(en)
|
2019-07-15 |
2020-05-19 |
Vigene Biosciences Inc. |
Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
|
US10557149B1
(en)
*
|
2019-07-15 |
2020-02-11 |
Vigene Biosciences, Inc. |
Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
|
JP2022544015A
(ja)
|
2019-07-23 |
2022-10-17 |
ユニバーシティ オブ ロチェスター |
CRISPR-Casでの標的化されたRNA切断
|
US20220396806A1
(en)
|
2019-07-26 |
2022-12-15 |
Akouos, Inc. |
Methods of treating hearing loss using a secreted target protein
|
JP2022544004A
(ja)
|
2019-07-26 |
2022-10-17 |
リジェネックスバイオ インコーポレイテッド |
操作された核酸調節エレメントならびにその使用方法
|
RU2751592C2
(ru)
*
|
2019-08-22 |
2021-07-15 |
Общество С Ограниченной Ответственностью "Анабион" |
Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
|
KR20220051246A
(ko)
|
2019-08-26 |
2022-04-26 |
리젠엑스바이오 인크. |
완전 인간 번역 후 변형된 항-VEGF Fab를 사용한 당뇨병성 망막병증의 치료
|
CA3149449A1
(en)
|
2019-09-19 |
2021-03-25 |
Evelyne GICQUEL |
Gene therapy expression system alleviating cardiac toxicity of fkrp
|
WO2021067598A1
(en)
|
2019-10-04 |
2021-04-08 |
Ultragenyx Pharmaceutical Inc. |
Methods for improved therapeutic use of recombinant aav
|
WO2021071835A1
(en)
|
2019-10-07 |
2021-04-15 |
Regenxbio Inc. |
Adeno-associated virus vector pharmaceutical composition and methods
|
JP2022551483A
(ja)
|
2019-10-08 |
2022-12-09 |
トラスティーズ オブ ボストン カレッジ |
複数の異なる非天然アミノ酸を含有するタンパク質、並びにそのようなタンパク質の製造方法及び使用方法
|
MX2022004352A
(es)
|
2019-10-17 |
2022-07-19 |
Stridebio Inc |
Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
|
JP2022552014A
(ja)
|
2019-10-18 |
2022-12-14 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
Irf2bpl遺伝子の変異に関連する障害の治療のための材料および方法
|
EP4055030A4
(de)
*
|
2019-11-08 |
2024-05-22 |
Harvard College |
Virale kapsidpolypeptide
|
US20230016983A1
(en)
|
2019-11-19 |
2023-01-19 |
lNSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) |
Antisense oligonucleotides and thier use for the treatment of cancer
|
JP2023502474A
(ja)
|
2019-11-22 |
2023-01-24 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
Ighmbp2遺伝子に関連する障害の治療のための材料および方法
|
CN115023242A
(zh)
*
|
2019-11-22 |
2022-09-06 |
费城儿童医院 |
腺相关病毒载体变体
|
TW202134260A
(zh)
|
2019-11-28 |
2021-09-16 |
美商銳進科斯生物股份有限公司 |
微小肌縮蛋白基因療法之構築體及其用途
|
EP4072595A1
(de)
|
2019-12-10 |
2022-10-19 |
Takeda Pharmaceutical Company Limited |
Adeno-assoziierte virusvektoren zur behandlung der hunter-krankheit
|
US20230227515A1
(en)
|
2019-12-20 |
2023-07-20 |
Research Institute At Nationwide Children's Hospital |
Optimized gene therapy for targeting muscle in muscle diseases
|
TW202140791A
(zh)
|
2020-01-13 |
2021-11-01 |
美商霍蒙拉奇醫藥公司 |
治療苯酮尿症之方法
|
IL294625A
(en)
|
2020-01-22 |
2022-09-01 |
Regenxbio Inc |
Treatment of mucopolysaccharidosis i with human fully glycosylated human alpha-l-iduronidase (idua)
|
JP2023512043A
(ja)
|
2020-01-29 |
2023-03-23 |
レジェンクスバイオ インコーポレーテッド |
ムコ多糖症ivaの治療
|
JP2023512242A
(ja)
|
2020-01-29 |
2023-03-24 |
レジェンクスバイオ インコーポレーテッド |
ヒト神経細胞またはヒトグリア細胞が産生した組換えヒトイズロン酸-2-スルファターゼ(ids)によるムコ多糖症iiの治療
|
WO2021158982A2
(en)
|
2020-02-07 |
2021-08-12 |
University Of Rochester |
Targeted translation of rna with crispr-cas13 to enhance protein synthesis
|
KR20220141829A
(ko)
|
2020-02-07 |
2022-10-20 |
유니버시티 오브 로체스터 |
리보자임-매개 rna 조립 및 발현
|
JP2023514204A
(ja)
|
2020-02-14 |
2023-04-05 |
ウルトラジェニックス ファーマシューティカル インコーポレイテッド |
Cdkl5欠損障害を処置するための遺伝子治療
|
AU2021230476A1
(en)
|
2020-03-02 |
2022-10-20 |
Tenaya Therapeutics, Inc. |
Gene vector control by cardiomyocyte-expressed microRNAs
|
CN115484978A
(zh)
|
2020-03-05 |
2022-12-16 |
尼奥克斯医疗有限公司 |
使用免疫细胞治疗癌症的方法和组合物
|
WO2021188892A1
(en)
|
2020-03-19 |
2021-09-23 |
Ultragenyx Pharmaceutical Inc. |
Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
|
US20240024520A1
(en)
|
2020-03-27 |
2024-01-25 |
UCB Biopharma SRL |
Autonomous knob domain peptides
|
JP2023519307A
(ja)
|
2020-03-27 |
2023-05-10 |
ユニバーシティ オブ ロチェスター |
Crispr-cas13によるウイルスrnaの標的化破壊
|
JP2023519344A
(ja)
|
2020-03-27 |
2023-05-10 |
ユニバーシティ オブ ロチェスター |
CRISPR-Cas13 crRNAアレイ
|
WO2021202532A1
(en)
|
2020-03-31 |
2021-10-07 |
Ultragenyx Pharmaceutical Inc. |
Gene therapy for treating propionic acidemia
|
WO2021207636A2
(en)
|
2020-04-10 |
2021-10-14 |
Sola Biosciences Llc |
Compositions and methods for the treatment of protein aggregation disorders
|
TW202208626A
(zh)
|
2020-04-24 |
2022-03-01 |
美商生命編輯公司 |
Rna引導核酸酶及其活性片段與變體,以及使用方法
|
CA3179895A1
(en)
|
2020-04-24 |
2021-10-28 |
Inserm (Institut National De La Sante Et De La Recherche Medicale) |
Methods for preventing induction of immune responses to the transduced cells expressing a transgene product after ocular gene therapy
|
EP4143215A2
(de)
|
2020-04-28 |
2023-03-08 |
SOLA Biosciences LLC |
Zusammensetzungen und verfahren zur behandlung von tdp-43-proteinopathien
|
EP4143216A1
(de)
|
2020-04-29 |
2023-03-08 |
Bristol-Myers Squibb Company |
Miniaturisierte dystrophine mit spektrinfusionsdomänen und verwendungen davon
|
MX2022014258A
(es)
|
2020-05-12 |
2023-02-22 |
Univ Pennsylvania |
Composiciones para la reducción específica de un fármaco de la expresión de transgén.
|
WO2021231538A2
(en)
|
2020-05-13 |
2021-11-18 |
Akouos, Inc. |
Compositions and methods for treating kcnq4-associated hearing loss
|
CA3183171A1
(en)
|
2020-05-13 |
2021-11-18 |
Akouos, Inc. |
Compositions and methods for treating slc26a4-associated hearing loss
|
WO2021236908A2
(en)
|
2020-05-20 |
2021-11-25 |
Biomarin Pharmaceutical Inc. |
Use of regulatory proteins for the production of adeno-associated virus
|
WO2021248038A1
(en)
|
2020-06-05 |
2021-12-09 |
Sola Biosciences Llc |
Compositions and methods for the treatment of synucleinopathies
|
BR112022025586A2
(pt)
|
2020-06-15 |
2023-03-07 |
Res Inst Nationwide Childrens Hospital |
Liberação de vetor de vírus adenoassociado para distrofias musculares
|
TW202214695A
(zh)
|
2020-06-17 |
2022-04-16 |
賓州大學委員會 |
用於治療基因療法患者之組成物及方法
|
EP4168052A2
(de)
|
2020-06-19 |
2023-04-26 |
Genethon |
Gentherapie-expressionssystem, das eine adequate expression in den muskeln und im herzen von sgcg ermöglicht
|
JP2023540429A
(ja)
|
2020-07-10 |
2023-09-25 |
アンセルム(アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル) |
てんかんを治療するための方法及び組成物
|
US20230277686A1
(en)
|
2020-07-10 |
2023-09-07 |
Genethon |
A novel muscle-specific promoter
|
TW202216244A
(zh)
|
2020-07-13 |
2022-05-01 |
賓州大學委員會 |
有用於治療夏馬杜三氏病之組成物
|
AU2021307447A1
(en)
|
2020-07-15 |
2023-03-09 |
University Of Rochester |
Targeted RNA cleavage with dCas13-RNase fusion proteins
|
US20220145328A1
(en)
|
2020-07-30 |
2022-05-12 |
Shape Therapeutics Inc. |
STABLE CELL LINES FOR INDUCIBLE PRODUCTION OF rAAV VIRIONS
|
IL300410A
(en)
|
2020-08-14 |
2023-04-01 |
Univ Pennsylvania |
Novel AAV capsids and compositions containing them
|
MX2023002293A
(es)
|
2020-08-24 |
2023-05-19 |
Univ Pennsylvania |
Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica.
|
JP2023541444A
(ja)
|
2020-09-15 |
2023-10-02 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
筋ジストロフィー患者における多様なdmd変異の補正のためのaav媒介性の相同性非依存的標的化組み込み遺伝子編集
|
EP4214242A1
(de)
|
2020-09-15 |
2023-07-26 |
RegenxBio Inc. |
Vektorisierte antikörper für antivirale therapie
|
WO2022060915A1
(en)
|
2020-09-15 |
2022-03-24 |
Regenxbio Inc. |
Vectorized lanadelumab and administration thereof
|
US20230383278A1
(en)
|
2020-09-18 |
2023-11-30 |
The United States Of America,As Represented By The Secretary,Department Of Health And Human Services |
Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
|
AU2021349277A1
(en)
|
2020-09-28 |
2023-05-11 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
US20220098615A1
(en)
|
2020-09-30 |
2022-03-31 |
NGGT, Inc. |
Dual functional expression vectors and methods of use thereof
|
US20240024508A1
(en)
|
2020-10-07 |
2024-01-25 |
Regenxbio Inc. |
Formulations for suprachoroidal administration such as high viscosity formulations
|
WO2022076595A1
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Formulations for suprachoroidal administration such as gel formulations
|
CA3197342A1
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Gene therapy for ocular manifestations of cln2 disease
|
WO2022076591A1
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Formulations for suprachoroidal administration such as formulations with aggregate formation
|
WO2022076750A2
(en)
|
2020-10-07 |
2022-04-14 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns or muscle delivery
|
AU2021356667A1
(en)
|
2020-10-07 |
2023-06-08 |
Regenxbio Inc. |
Adeno-associated viruses for ocular delivery of gene therapy
|
WO2022076803A1
(en)
|
2020-10-09 |
2022-04-14 |
The Trustees Of The University Of Pennsylvania |
Compositions and methods for treatment of fabry disease
|
US11781156B2
(en)
|
2020-10-09 |
2023-10-10 |
Tenaya Therapeutics, Inc. |
Plakophillin-2 gene therapy methods and compositions
|
CA3195967A1
(en)
|
2020-10-28 |
2022-05-05 |
Xu Wang |
Vectorized anti-tnf-? antibodies for ocular indications
|
WO2022094157A1
(en)
|
2020-10-28 |
2022-05-05 |
Regenxbio Inc. |
Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
|
WO2022094255A2
(en)
|
2020-10-29 |
2022-05-05 |
Regenxbio Inc. |
Vectorized factor xii antibodies and administration thereof
|
EP4237453A1
(de)
|
2020-10-29 |
2023-09-06 |
RegenxBio Inc. |
Vektorisierte tnf-alpha antagonisten für augenindikationen
|
MX2023005113A
(es)
|
2020-10-29 |
2023-08-04 |
Univ Pennsylvania |
Cápsides de aav y composiciones que las contienen.
|
EP4237545A1
(de)
|
2020-11-02 |
2023-09-06 |
BioMarin Pharmaceutical Inc. |
Verfahren zur anreicherung von adeno-assoziiertem virus
|
US20240026356A1
(en)
|
2020-11-30 |
2024-01-25 |
Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd)
|
EP4256065A2
(de)
|
2020-12-01 |
2023-10-11 |
The Trustees of The University of Pennsylvania |
Neue zusammensetzungen mit gewebespezifischen zielmotiven und zusammensetzungen damit
|
IL303317A
(en)
|
2020-12-01 |
2023-07-01 |
Akouos Inc |
ANTI-NATURAL ANTIBODY STRUCTURES AND RELATED METHODS FOR THE TREATMENT OF SYMPTOMS ASSOCIATED WITH VESTIBULAR SWANNOMA
|
JP2023551911A
(ja)
|
2020-12-01 |
2023-12-13 |
ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア |
アンジェルマン症候群の治療のための組成物及びその使用
|
WO2022122733A1
(en)
|
2020-12-08 |
2022-06-16 |
Genethon |
New gene therapy for the treatment of duchenne muscular dystrophy
|
CA3201743A1
(en)
|
2020-12-16 |
2022-06-23 |
Robert STADELMAN |
Method of producing a recombinant adeno-associated virus particle
|
TW202241943A
(zh)
|
2020-12-29 |
2022-11-01 |
美商銳進科斯生物股份有限公司 |
Tau特異性抗體基因療法組合物、方法及其用途
|
PE20240012A1
(es)
|
2020-12-29 |
2024-01-04 |
Akouos Inc |
Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1
|
AU2022206197A1
(en)
|
2021-01-05 |
2023-07-13 |
Selecta Biosciences, Inc. |
Viral vector dosing protocols
|
CN116848255A
(zh)
|
2021-01-21 |
2023-10-03 |
再生生物股份有限公司 |
改进的重组多肽和病毒的生产
|
WO2022164860A1
(en)
|
2021-01-27 |
2022-08-04 |
Research Institute At Nationwide Children's Hospital |
Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
|
MX2023008826A
(es)
|
2021-02-01 |
2023-09-15 |
Regenxbio Inc |
Terapia génica para lipofuscinosis neuronal ceroidea.
|
AU2022216260A1
(en)
|
2021-02-03 |
2023-08-17 |
Research Institute At Nationwide Children's Hospital |
Compositions and methods for treating disease associated with dux4 overexpression
|
WO2022173605A2
(en)
|
2021-02-10 |
2022-08-18 |
Regenxbio Inc. |
Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
|
WO2022183052A1
(en)
|
2021-02-26 |
2022-09-01 |
Takeda Pharmaceutical Company Limited |
Composition and methods for the treatment of fabry disease
|
WO2022187571A1
(en)
|
2021-03-04 |
2022-09-09 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
|
EP4308711A1
(de)
|
2021-03-19 |
2024-01-24 |
Adrenas Therapeutics, Inc. |
Gentherapien für 21-hydroxylase-mangel
|
CA3215141A1
(en)
|
2021-04-12 |
2022-10-20 |
James M. Wilson |
Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
|
CA3216711A1
(en)
|
2021-04-13 |
2022-10-20 |
Research Institute At Nationwide Children's Hospital |
Recombinant adeno-associated virus encoding methyl-cpg binding protein 2 for treating pitt hopkins syndrome via intrathecal delivery
|
JP2024515715A
(ja)
|
2021-04-23 |
2024-04-10 |
ユニバーシティ オブ ロチェスター |
レトロウイルスインテグラーゼ-Cas融合タンパク質を使用した指向性非相同DNA挿入によるゲノム編集及び治療の方法
|
CA3216004A1
(en)
|
2021-04-23 |
2022-10-27 |
The Trustees Of The University Of Pennsylvania |
Novel compositions with brain-specific targeting motifs and compositions containing same
|
CA3217491A1
(en)
|
2021-04-23 |
2022-10-27 |
Research Institute At Nationwide Children's Hospital |
Products and methods for treating muscular dystrophy
|
CA3216744A1
(en)
|
2021-04-26 |
2022-11-03 |
Regenxbio Inc. |
Microdystrophin gene therapy administration for treatment of dystrophinopathies
|
TW202309066A
(zh)
|
2021-04-27 |
2023-03-01 |
賓州大學委員會 |
衍生自豬的腺相關病毒衣殼及其用途
|
WO2022229703A2
(en)
|
2021-04-30 |
2022-11-03 |
Takeda Pharmaceutical Company, Ltd. |
New aav8 based immune escaping variants
|
EP4330270A2
(de)
|
2021-04-30 |
2024-03-06 |
Takeda Pharmaceutical Company Limited |
Aav8-capsid-varianten mit verbessertem leber-targeting
|
WO2022235614A2
(en)
|
2021-05-04 |
2022-11-10 |
Regenxbio Inc. |
Novel aav vectors and methods and uses thereof
|
WO2022241030A1
(en)
|
2021-05-11 |
2022-11-17 |
Regenxbio Inc. |
Treatment of duchenne muscular dystrophy and combinations thereof
|
AU2022279062A1
(en)
|
2021-05-17 |
2024-01-04 |
Sarepta Therapeutics, Inc. |
Production of recombinant aav vectors for treating muscular dystrophy
|
EP4108263A3
(de)
|
2021-06-02 |
2023-03-22 |
Research Institute at Nationwide Children's Hospital |
Rekombinante adeno-assoziierte virusprodukte und verfahren zur behandlung von gliedergürtel-muskeldystrophie 2a
|
AU2022299552A1
(en)
*
|
2021-06-25 |
2024-01-04 |
Oxford Biomedica (Us) Llc |
Adeno-associated virus packaging systems
|
EP4366757A1
(de)
|
2021-07-07 |
2024-05-15 |
INSERM (Institut National de la Santé et de la Recherche Médicale) |
Synergistische kombination von rdcfv2 und rdcvf2l zur behandlung von tauopathien
|
IL309742A
(en)
|
2021-07-08 |
2024-02-01 |
Tenaya Therapeutics Inc |
Optimal expression cassettes for gene therapy
|
CA3226452A1
(en)
|
2021-07-19 |
2023-01-26 |
New York University |
Auf1 combination therapies for treatment of muscle degenerative disease
|
TW202328446A
(zh)
|
2021-08-11 |
2023-07-16 |
美商堅固生物科技公司 |
肌肉萎縮症之治療
|
AR126840A1
(es)
*
|
2021-08-20 |
2023-11-22 |
Llc «Anabion» |
Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 5 (aav5), cápside y vector basado en esta
|
AR126839A1
(es)
*
|
2021-08-20 |
2023-11-22 |
Llc «Anabion» |
Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
|
WO2023056329A1
(en)
|
2021-09-30 |
2023-04-06 |
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Compositions and methods for treating kcnq4-associated hearing loss
|
WO2023056399A1
(en)
|
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2023-04-06 |
The Trustees Of The University Of Pennsylvania |
Novel aav capsids and compositions containing same
|
WO2023060113A1
(en)
|
2021-10-05 |
2023-04-13 |
Regenxbio Inc. |
Compositions and methods for recombinant aav production
|
WO2023060269A1
(en)
|
2021-10-07 |
2023-04-13 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for targeted delivery
|
WO2023060272A2
(en)
|
2021-10-07 |
2023-04-13 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns tropic delivery
|
CA3234702A1
(en)
|
2021-10-07 |
2023-04-13 |
Research Institute At Nationwide Children's Hospital |
Products and methods for myelin protein zero silencing and treating cmt1b disease
|
WO2023060221A2
(en)
|
2021-10-08 |
2023-04-13 |
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Compositions and methods for the treatment of proteopathies
|
CA3234720A1
(en)
|
2021-10-08 |
2023-04-13 |
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Compositions and methods for the treatment of p53-mediated cancers
|
WO2023064367A1
(en)
|
2021-10-12 |
2023-04-20 |
Selecta Biosciences, Inc. |
Methods and compositions for attenuating anti-viral transfer vector igm responses
|
CA3235145A1
(en)
|
2021-10-12 |
2023-04-20 |
Bridgebio Gene Therapy Llc |
Methods and compositions for treating leukodystrophies
|
CA3235269A1
(en)
|
2021-10-12 |
2023-04-20 |
Cartesian Therapeutics, Inc. |
Viral vector dosing protocols
|
EP4219726A1
(de)
|
2021-10-15 |
2023-08-02 |
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Selbstkomplementärer adeno-assoziierter virusvektor und dessen verwendung bei der behandlung von muskeldystrophie
|
WO2023077092A1
(en)
|
2021-10-28 |
2023-05-04 |
Regenxbio Inc. |
Engineered nucleic acid regulatory elements and methods and uses thereof
|
CA3237037A1
(en)
|
2021-11-14 |
2023-05-19 |
Cartesian Therapeutics, Inc. |
Multiple dosing with viral vectors
|
WO2023087019A2
(en)
|
2021-11-15 |
2023-05-19 |
The Trustees Of The University Of Pennsylvania |
Compositions for drg-specific reduction of transgene expression
|
JP2023081369A
(ja)
|
2021-11-30 |
2023-06-09 |
リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル |
自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
|
WO2023102517A1
(en)
|
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The Trustees Of The University Of Pennsylvania |
Compositions and methods for treatment of fabry disease
|
WO2023122669A1
(en)
|
2021-12-21 |
2023-06-29 |
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Materials and methods for the treatment of limb girdle muscular dystrophy
|
TW202340467A
(zh)
|
2022-01-10 |
2023-10-16 |
賓州大學委員會 |
有用於治療c9orf72介導之病症之組成物及方法
|
WO2023139557A1
(en)
|
2022-01-24 |
2023-07-27 |
LifeEDIT Therapeutics, Inc. |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
EP4215614A1
(de)
|
2022-01-24 |
2023-07-26 |
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Kombinationstherapie für dystrophin-verwandte krankheiten
|
TW202342525A
(zh)
|
2022-02-02 |
2023-11-01 |
美商阿科奧斯公司 |
用於治療前庭神經鞘瘤相關症狀之抗vegf抗體構築體及相關方法
|
WO2023168400A2
(en)
|
2022-03-03 |
2023-09-07 |
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Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
|
WO2023172624A1
(en)
|
2022-03-09 |
2023-09-14 |
Selecta Biosciences, Inc. |
Immunosuppressants in combination with anti-igm agents and related dosing
|
TW202346590A
(zh)
|
2022-03-13 |
2023-12-01 |
美商銳進科斯生物股份有限公司 |
經修飾之肌肉特異性啟動子
|
WO2023178220A1
(en)
|
2022-03-16 |
2023-09-21 |
Regenxbio Inc. |
Compositions and methods for recombinant aav production
|
WO2023182476A1
(ja)
*
|
2022-03-25 |
2023-09-28 |
学校法人自治医科大学 |
アデノ随伴ウイルス(aav)ベクターの効率の良い産生システム
|
WO2023183623A1
(en)
|
2022-03-25 |
2023-09-28 |
Regenxbio Inc. |
Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
|
WO2023187728A1
(en)
|
2022-04-01 |
2023-10-05 |
Takeda Pharmaceutical Company Limited |
Gene therapy for diseases with cns manifestations
|
TW202404651A
(zh)
|
2022-04-06 |
2024-02-01 |
美商銳進科斯生物股份有限公司 |
用於脈絡膜上投與之調配物諸如形成聚集體之調配物
|
WO2023196892A1
(en)
|
2022-04-06 |
2023-10-12 |
The Trustees Of The University Of Pennsylvania |
Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
|
WO2023196873A1
(en)
|
2022-04-06 |
2023-10-12 |
Regenxbio Inc. |
Pharmaceutical composition comprising a recombinant adeno-associated virus vector with an expression cassette encoding a transgene forsuprachoidal administration
|
TW202345913A
(zh)
|
2022-04-06 |
2023-12-01 |
美商銳進科斯生物股份有限公司 |
用於脈絡膜上投與之調配物諸如凝膠調配物
|
WO2023201207A1
(en)
|
2022-04-11 |
2023-10-19 |
Tenaya Therapeutics, Inc. |
Adeno-associated virus with engineered capsid
|
WO2023201277A1
(en)
|
2022-04-14 |
2023-10-19 |
Regenxbio Inc. |
Recombinant adeno-associated viruses for cns tropic delivery
|
WO2023201308A1
(en)
|
2022-04-14 |
2023-10-19 |
Regenxbio Inc. |
Gene therapy for treating an ocular disease
|
WO2023205610A2
(en)
|
2022-04-18 |
2023-10-26 |
Regenxbio Inc. |
Hybrid aav capsids
|
WO2023215807A1
(en)
|
2022-05-03 |
2023-11-09 |
Regenxbio Inc. |
VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS
|
TW202400803A
(zh)
|
2022-05-03 |
2024-01-01 |
美商銳進科斯生物股份有限公司 |
載體化抗補體抗體與補體劑及其投與
|
WO2023214346A1
(en)
|
2022-05-06 |
2023-11-09 |
Novartis Ag |
Novel recombinant aav vp2 fusion polypeptides
|
WO2023240177A1
(en)
|
2022-06-08 |
2023-12-14 |
Research Instiitute At Nationwide Children's Hospital |
Products and methods for treating diseases or conditions associated with mutant or pathogenic kcnq3 expression
|
WO2023239627A2
(en)
|
2022-06-08 |
2023-12-14 |
Regenxbio Inc. |
Methods for recombinant aav production
|
WO2024015966A2
(en)
|
2022-07-15 |
2024-01-18 |
The Trustees Of The University Of Pennsylvania |
Recombinant aav having aav clade d and clade e capsids and compositions containing same
|
WO2024017990A1
(en)
|
2022-07-21 |
2024-01-25 |
Institut National de la Santé et de la Recherche Médicale |
Methods and compositions for treating chronic pain disorders
|
WO2024036324A1
(en)
|
2022-08-11 |
2024-02-15 |
Selecta Biosciences, Inc. |
Compositions and methods related to immunoglobulin proteases and fusions thereof
|
WO2024033901A1
(en)
|
2022-08-12 |
2024-02-15 |
LifeEDIT Therapeutics, Inc. |
Rna-guided nucleases and active fragments and variants thereof and methods of use
|
WO2024044725A2
(en)
|
2022-08-24 |
2024-02-29 |
Regenxbio Inc. |
Recombinant adeno-associated viruses and uses thereof
|
WO2024042485A1
(en)
|
2022-08-25 |
2024-02-29 |
Takeda Pharmaceutical Company Limited |
Composition for use in the treatment of fabry disease
|
WO2024064863A2
(en)
|
2022-09-22 |
2024-03-28 |
Biomarin Pharmaceutical Inc. |
Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
|
WO2024064856A1
(en)
|
2022-09-22 |
2024-03-28 |
Biomarin Pharmaceutical Inc. |
Treatment of cardiomyopathy with aav gene therapy vectors
|
WO2024064913A1
(en)
|
2022-09-23 |
2024-03-28 |
Sarepta Therapeutics, Inc. |
Recombinant aav vectors for treating muscular dystrophy
|
WO2024073669A1
(en)
|
2022-09-30 |
2024-04-04 |
Regenxbio Inc. |
Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
|
WO2024081706A1
(en)
|
2022-10-11 |
2024-04-18 |
Research Institute At Nationwide Children's Hospital |
Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)
|
WO2024081746A2
(en)
|
2022-10-11 |
2024-04-18 |
Regenxbio Inc. |
Engineered nucleic acid regulatory elements and methods and uses thereof
|